CIRM Board

State Stem Cell & Gene Therapy Agency Sets up Support Program to Help Patients Participate in Clinical Trials

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For many patients battling deadly diseases, getting access to a clinical trial can be life-saving, but it can also be very challenging. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved a concept plan to make it financially and logistically easier for patients to take part in CIRM-funded clinical trials.

The plan will create a Patient Support Program (PSP) to provide support to California patients being evaluated or enrolled in CIRM-supported clinical trials, with a particular emphasis on helping underserved populations.

“Helping scientists develop stem cell and gene therapies is just part of what we do at CIRM. If those clinical trials and resulting therapies are not accessible to the people of California, who are making all this possible, then we have not fulfilled our mission.” says Maria T. Millan, M.D., President and CEO of CIRM.

The Patient Support Plan will offer a range of services including:

  • Clinical trial navigation, directing patients to appropriate CIRM-supported clinical trials.
  • Logistical support for patients being evaluated or enrolled in clinical trials.
  • Financial support for under resourced and underserved populations in CIRM-supported clinical trials, including the CIRM Patient Assistance Fund (PAF).  This support includes transportation/travel expenses, such as gasoline, tolls, parking, airfare, taxi, train, lodging, and meals during travel.
  • Providing nurse navigator support for the psychosocial, emotional, and practical needs of patients and their families.

The funds for the PSP are set aside under Proposition 14, the voter-approved initiative that re-funded CIRM in 2020. Under Prop 14 CIRM money that CIRM grantees earn from licensing, inventions or technologies is to be spent “offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.”

Currently, the CIRM Licensing Revenues and Royalties Fund has a balance of $15.6 million derived from royalty payments.

“The patient support program and financial resources will not only help patients in need, it will also help increase the likelihood that these clinical trials will succeed,” says Sean Turbeville, Ph.D., Vice President of Medical Affairs and Policy at CIRM. “We know cell and gene therapies can be particularly challenging for patients and their families. The financial challenges, the long-distance traveling, extended evaluation, and family commitments can make it difficult to enroll and retain patients. The aim of the PSP is to change that.”

The overall objective of this funding opportunity is to establish a statewide program that, over five years, is expected to support hundreds of patients in need as they participate in the growing number of CIRM-supported clinical trials. The program is expected to cost between $300,000 to $500,000 a year. That money will come from the Medical Affairs budget and not out of the patient assistance fund.

The first phase of the program will identify an organization, through a competitive process, that has the expertise to provide patient support services including:

  • Maintaining a call and support center.
  • Assessing patient eligibility for financial assistance.
  • Reporting to CIRM on patients needs and center performance

 You can find more information about the Patient Support Program on our website here and here.

So far, some encouraging news for stem cell clinical trial treating epilepsy

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Neurona Therapeutics is testing a new therapy for a drug-resistant form of epilepsy and has just released some encouraging early findings. The first patient treated went from having more than 30 seizures a month to just four seizures over a three-month period.

This clinical trial, funded by the California Institute for Regenerative Medicine (CIRM), is targeting  mesial temporal lobe epilepsy (MTLE), one of the most common forms of epilepsy. Because the seizures caused by MTLE are frequent, they can be particularly debilitating and increase the risk of a decreased quality of life, depression, anxiety and memory impairment.

Neurona’s therapy, called NRTX-1001, consists of a specialized type of neuronal cell derived from embryonic stem cells.  Neuronal cells are messenger cells that transmit information between different areas of the brain, and between the brain and the rest of the nervous system.

NRTX-1001 is injected into the brain in the area affected by the seizures where it releases neurotransmitters or chemical messengers that will block the signals in the brain causing the epileptic seizures.

The first patient treated had a nine-year history of epilepsy and, despite being on anti-epileptic medications, was experiencing dozens of seizures a month. Since the therapy he has had only four seizures in three months. The therapy hasn’t produced any serious side effects.

In a news release Dr. Cory Nicholas, Neurona’s President and CEO, said while this is only one patient, it’s good news.

“The reduced number of seizures reported by the first person to receive NRTX-1001 is very encouraging, and we remain cautiously optimistic that this reduction in seizure frequency will continue and extend to others entering this cell therapy trial. NRTX-1001 administration has been well tolerated thus far in the clinic, which is in line with the extensive preclinical safety data collected by the Neurona team. With recent clearance from the Data Safety Monitoring Board we are excited to continue patient enrollment. We are very grateful to these first participants, and thank the clinical teams for the careful execution of this pioneering study.”

CIRM has been a big supporter of this work from the early Discovery stage work to this clinical trial. That’s because when we find something promising, we want to do everything we can to help it live up to its promise.

Funding a Clinical Trial for a Functional Cure for HIV

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The use of antiretroviral drugs has turned HIV/AIDS from a fatal disease to one that can, in many cases in the US, be controlled. But these drugs are not a cure. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) voted to approve investing $6.85 million in a therapy that aims to cure the disease.

This is the 82nd clinical trial funded by CIRM.

There are approximately 38 million people worldwide living with HIV/AIDS. And each year there are an estimated 1.5 million new cases. The vast majority of those living with HIV do not have access to the life-saving antiretroviral medications that can keep the virus under control. People who do have access to the medications face long-term complications from them including heart disease, bone, liver and kidney problems, and changes in metabolism.

The antiretroviral medications are effective at reducing the viral load in people with HIV, but they don’t eliminate it. That’s because the virus that causes AIDS can integrate its DNA into long-living cells in the body and remain dormant. When people stop taking their medications the virus is able to rekindle and spread throughout the body.

Dr. William Kennedy and the team at Excision Bio Therapeutics have developed a therapeutic candidate called EBT-101. This is the first clinical study using the CRISPR-based platform for genome editing and excision of the latent form of HIV-1, the most common form of the virus that causes AIDS in the US and Europe. The goal is to eliminate or sufficiently reduce the hidden reservoirs of virus in the body to the point where the individual is effectively cured.

“To date only a handful of people have been cured of HIV/AIDS, so this proposal of using gene editing to eliminate the virus could be transformative,” says Dr. Maria Millan, President and CEO of CIRM. “In California alone there are almost 140,000 people living with HIV. HIV infection continues to disproportionately impact marginalized populations, many of whom are unable to access the medications that keep the virus under control. A functional cure for HIV would have an enormous impact on these communities, and others around the world.”

In a news release announcing they had dosed the first patient, Daniel Dornbusch, CEO of Excision, called it a landmark moment. “It is the first time a CRISPR-based therapy targeting an infectious disease has been administered to a patient and is expected to enable the first ever clinical assessment of a multiplexed, in vivo gene editing approach. We were able to reach this watershed moment thanks to years of innovative work by leading scientists and physicians, to whom we are immensely grateful. With this achievement, Excision has taken a major step forward in developing a one-time treatment that could transform the HIV pandemic by freeing affected people from life-long disease management and the stigma of disease.”

The Excision Bio Therapeutics team also scored high on their plan for Diversity, Equity and Inclusion. Reviewers praised them for adding on a partnering organization to provide commitments to serve underserved populations, and to engaging a community advisory board to help guide their patient recruitment.

CIRM has already invested almost $81 million in 20 projects targeting HIV/AIDS, including four clinical trials.

How stem cells helped Veronica fight retinitis pigmentosa and regain her vision

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Veronica and Elliott

Growing up Veronica McDougall thought everyone saw the world the way she did; blurry, slightly out-of-focus and with tunnel vision.  As she got older her sight got worse and even the strongest prescription glasses didn’t help. When she was 15 her brother tried teaching her to drive. One night she got into the driver’s seat to practice and told him she couldn’t see anything. Everything was just black. After that she stopped driving.   

Veronica says high school was really hard for her, but she managed to graduate and go to community college. As her vision deteriorated, she found it was increasingly hard to read the course work and impossible to see the assignments on the blackboard. Veronica says she was lucky to have some really supportive teachers — including the now First Lady Jill Biden — but eventually she had to drop out.  

Getting a diagnosis

When she was 24, she went to see a specialist who told her she had retinitis pigmentosa, a rare degenerative condition that would eventually leave her legally blind. She says it felt like a death sentence. “All of my dreams of becoming a nurse, of getting married, of having children, of traveling – it all just shattered in that moment.” 

Veronica says she went from being a happy, positive person to an angry depressed one. She woke up each morning terrified, wondering, “Is this the day I go blind?” 

Then her mother learned about a CIRM-funded clinical trial with a company called jCyte. Veronica applied to be part of it, was accepted and was given an injection of stem cells in her left eye. She says over the course of a few weeks, her vision steadily improved. 

“About a month after treatment, I was riding in the car with my mom and suddenly, I realized I could see her out of the corner of my eye while looking straight ahead. That had never, ever happened to me before. Because, I had been losing my peripheral vision at a young age without realizing that until up to that point, I had never had that experience.” 

A second chance at life

She went back to college, threw herself into her studies, started hiking and being more active. She says it was as if she was reborn. But in her senior year, just as she was getting close to finishing her degree, her vision began to deteriorate again. Fortunately, she was able to take part in a second clinical trial, and this time her vision came back stronger than ever. 

“I’m so grateful to the researchers who gave me my sight back with the treatment they have worked their entire lives to develop. I am forever grateful for the two opportunities to even receive these two injections and to be a part of an amazing experience to see again. I feel so blessed! Thank you for giving me my life back.” 

And in getting her life back, Veronica had a chance to give life. When she was at college she met and starting dating Robert, the man who was to become her partner. They now have a little boy, Elliott.  

As for the future, Veronica hopes to get a second stem cell therapy to improve her vision even further. Veronica’s two treatments were in her left eye. She is hoping that the Food and Drug Administration will one day soon approve jCyte’s therapy, so that she can get the treatment in her right eye. Then, she says, she’ll be able to see the world as the rest of us can.  

CIRM has invested more than $150 million in programs targeting vision loss, including four clinical trials for retinitis pigmentosa

Stem Cell Agency Invests $46 Million in New Education Program

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CIRM Bridges students 2022. The CIRM Board approved funding for a program to help even more students advance a career in science.

The governing Board of the California Institute for Regenerative Medicine (CIRM) has approved $46,076,430 to invest in its newest education pillar- the COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem cell Science) training program.

Education is at the core of CIRM’s mission of accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world. And funding these additional programs is an important step in ensuring that California has a well-trained stem cell workforce.

The objective of COMPASS is to prepare a diverse cadre of undergraduate students for careers in regenerative medicine through combining hands-on research opportunities with strategic and structured mentorship experiences.

“Education and infrastructure are two funding pillars critical for creating the next generation of researchers and conducting stem cell based clinical trials,” says Jonathan Thomas, Ph.D., J.D., Chair of the CIRM Board. “The importance of these programs was acknowledged in Proposition 14 and we expect that they will continue to be important components of CIRM’s programs and strategic direction in the years to come.”

Most undergraduate research training programs, including those targeting students from underserved communities, target individuals with predefined academic credentials as well as a stated commitment towards graduate school, medical school, or faculty positions in academia. COMPASS will support the development and implementation of novel strategies to recognize and foster untapped talent that can lead to new and valuable perspectives that are specific to the challenges of regenerative medicine, and that will create new paths to a spectrum of careers that are not always apparent to students in the academic, undergraduate environment.

COMPASS will complement but not compete with CIRM’s Bridges program, a subset of which serve a different, but equally important population of undergraduate trainees; similarly, the program is unlikely to compete for the same pools of students that would be most likely to receive support through the major NIH Training Programs such as MARC and RISE.

Here are the 16 successful applicants.

Application numberTitlePrincipal InvestigatorAmount
EDUC5-13840  The COMPASS Scholars Program – Developing Today’s Untapped Talent into Tomorrow’s STEM Cell Researchers    John Matsui, University of California, Berkeley    $2,908,950
EDUC5-13634  COMPASS Undergraduate Program  Alice F Tarantal, University of California, Davis    $2,909,950  
EDUC5-13637  Research Mentorship Program in Regenerative Medicine Careers for a Diverse Undergraduate Student Body    Brian J. Cummings, University of California, Irvine    $2,729,900
EDUC5-13665  CIRM COMPASS Training Program (N-COMPASS)  Cindy S Malone, The University Corporation at California State University, Northridge    $2,909,700  
EDUC5-13817  COMPASS: Accelerating Stem Cell Research by Educating and Empowering New Stem Cell Researchers  Tracy L Johnson, University of California, Los Angeles    $2,910,000  
EDUC5-13744  Training and mentorship program in stem cell biology and engineering: A COMPASS for the future  Dennis Clegg, University of California, Santa Barbara    $2,746,000  
EDUC5-13636  Research Training and Mentorship Program to Inspire Diverse Undergraduates toward Regenerative Medicine
Careers (RAMP)		  Huinan Hannah Liu, The Regents of the University of California on behalf of its Riverside Campus    $2,910,000  
EDUC5-13679  Inclusive Pathways for a Stem Cell Scholar (iPSCs) Undergraduate Training Program    Lily Chen, San Francisco State University    $2,894,500
EDUC5-13733  A COMPASS to guide the growth of a diverse regenerative medicine workforce that represents California and benefits
the world		  Kristen OHalloran Cardinal, Cal Poly Corporation, an Auxiliary of California Polytechnic State University, San Luis Obispo    $2,887,939  
EDUC5-13619  Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences (IDEA-CBMS)  Michael Fino, MiraCosta College    $2,894,500  
EDUC5-13667  COMPASS Program for Southern California Hispanic Serving Institution  Bianca Romina Mothé, California State University San Marcos Corporation    $2,877,200  
EDUC5-13653  Student Pluripotency: Realizing Untapped Undergraduate Potential in Regenerative Medicine  Daniel Nickerson, California State University, San Bernardino    $2,909,853  
EDUC5-13647  COMPASS: an inclusive Pipeline for Research and Other Stem cell-based Professions in Regenerative medicine
(iPROSPR)  		  Alison Miyamoto, CSU Fullerton Auxiliary Services Corporation    $2,883,440
EDUC5-13686  Training Undergraduates in Stem Cell Engineering and Biology (TUSCEB)    Kara E McCloskey, University of California, Merced    $2,909,999
EDUC5-13853  COMPASS: Guiding Undergraduates to Careers in Regenerative Medicine    Senta Georgia, University of Southern California    $2,899,999
EDUC5-13910  IDEA-CBMS – Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences    James Dekloe, Solano Community College    $2,894,500

The researcher who is following her bliss, and tackling diseases of aging at the same time

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Dr. Jill Helms, and associate! Photo courtesy Stanford University

Jill Helms is not your average Stanford University faculty member. Yes, she is a professor in the Department of Surgery. Yes, she has published lots of scientific studies. Yes, she is a stem cell scientist (funded by CIRM). And yes, she is playing a leading role in Ankasa Regenerative Therapeutics, a company focused on tissue repair and regeneration. But she is so much more than all that.  

She is a brilliant public speaker, a fashionista, and has ridden her horse to work (well, Stanford is referred to as The Farm, so why not!) and she lives on a farm of her own called “Follow Your Bliss.” The name comes from philosopher Joseph Campbell who wrote, “If you follow your bliss, you put yourself on a kind of path that has been there all the while, waiting for you. And the life you ought to be living is the one you are living.”  

Dr. Helms says that pretty much sums up her life. She says she feels enormously blessed.  

Well, we felt enormously blessed when she agreed to sit down with us and chat about her work, her life and her love of fashion for the California Institute for Regenerative Medicine podcast, Talking ‘Bout (re)Generation.  

We hope you enjoy the latest episode! 

A grandmother’s legacy, a stem cell scientist

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Emily Smith, CIRM Bridges student

The California Institute for Regenerative (CIRM) has a number of education programs geared towards training the next generation of stem cell and gene therapy researchers. Each student comes to the program with their own motivation, their own reasons for wanting to be a scientist. This is Emily Smith’s story.

Surrounded by the cold white walls of a hospital room, my family suddenly found themselves on the other side of medicine. Void of any answers or cures, this new reality was full of doubt. As we witnessed assurance dwindle into a look of angst, the doctor’s lips stiffened as he faltered to say the words that would change my grandmother’s life forever. The spinal cancer they had gone in to extract was a misdiagnosed nothing. Instead, the exploration of his scalpel left her paralyzed from the chest down.

Seemingly simple day-to-day moments of my life became the building blocks of my passion for science today. Early realizations of the hurdles laced throughout my grandmother’s life. Vivid memories of my mother’s weary smile as she read articles on the newest advancements in stem cell research. Collectively, what these fragments of time nurtured was hope. I grew to have a dream that something different awaited us in the future. With purpose, I dove into the world of research as an undergraduate.

Today, I am a CIRM Bridges to Stem Cell Research Intern at the Sanford Consortium for Regenerative Medicine. I received my acceptance into the program about a month after my grandmother’s passing. She never saw a cure, let alone an effective treatment.

My position allows me to understand why stem cell research takes time. The road from the bench to the clinic is a painstakingly deliberate one. And although we seek reason and order from the world of science, what we often find is how imperfect it all can be. At its root, I found that research is truly a human endeavor. That is why, as scientists, we must grapple with our lack of knowledge and failures with humility.

CIRM’s programs that train tomorrow’s scientists, such as Bridges, are important because they do more than simply transfer over skills from one generation to the next. Over the next year, I get the valuable experience of working with scientists who share a common dream. They understand the urgency of their research, value the quality of their findings, and put patient needs first. This mentorship ensures that a sense of responsibility is carried on throughout this field.

I applied to this program because stem cell research gave my family the gift of hope. Now, on the other side of the wait, I wish to serve patients and families like my own. I am incredibly grateful to be a part of the Bridges program and I will devote the full extent of my knowledge towards the advancement of this field.

How CIRM contributed to City of Hope study helping man with HIV into long-term remission

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The news that a stem cell transplant at City of Hope helped a man with HIV go into long-term remission made banner headlines around the world. As it should. It’s a huge achievement, particularly as the 66-year-old man had been living with HIV since 1988.

What wasn’t reported was that work supported by the California Institute for Regenerative Medicine played a role in making that happen.

The Stem Cell Transplant

First the news. In addition to living with HIV the man was diagnosed with acute leukemia. Doctors at City of Hope found a donor who was not only a perfect match to help battle the patient’s leukemia, but the donor also had a rare genetic mutation that meant they were resistant to most strains of HIV.

In transplanting blood stem cells from the donor to the patient they were able to send both his leukemia and HIV into remission. The patient stopped taking all his antiretroviral medications 17 months ago and today has no detectable levels of HIV.

In a news release  City of Hope hematologist Ahmed Aribi, M.D., said the patient didn’t experience any serious complications after the procedure.

“This patient had a high risk for relapsing from AML [acute myeloid leukemia], making his remission even more remarkable and highlighting how City of Hope provides excellent care treating complicated cases of AML and other blood cancers.”

It’s a remarkable achievement and is only the fifth time that a patient with both HIV and leukemia has been put into remission after a transplant from an HIV-resistant donor.

CIRM’s Contribution

So, what does that have to do with CIRM? Well, CIRM’s Alpha Clinics Network helped City of Hope get this case approved by an Institutional Review Board (IRB) and also helped in collecting and shipping the donor blood. In addition, part of the Alpha Clinics team at University of California San Diego helped with the reservoir analysis of blood and gut biopsies to check for any remaining signs of HIV.

It’s a reminder that this kind of achievement is a team effort and CIRM is very good at creating and supporting teams. The Alpha Clinics Network is a perfect example. We created it because there was a need for a network of world-class medical facilities with the experience and expertise to deliver a whole new kind of therapy. The Network has been remarkably successful in doing that with more than 200 clinical trials, taking care of more than 1,000 patients, and treating more than 40 different diseases.

This year our Board approved expanding the number of these clinics to better serve the people of California.

While the role of the Alpha Clinics Network in helping this one patient may seem relatively small, it was also an important one. And we are certainly not stopping here. We have invested more than $79 million in 19 different projects targeting HIV/AIDS, include four clinical trials.

We are in this for the long term and results like the man who had HIV and is now in remission are a sign we are heading in the right direction.

Stem Cell Agency funds clinical trial targeting scarred urethras

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A urethral stricture is scarring of the tube that carries urine out of the body. If left untreated it can be intensely painful and lead to kidney stones and infections. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) is investing more than $3.8 million in a Phase 1 clinical trial to create a stem cell-based therapy for the condition.

This is the 81st clinical trial that CIRM has funded.

When a scar, or stricture, forms along the urethra it impedes the flow of urine and causes other complications. James Yoo, M.D., Ph.D., and his team at Wake Forest University Health Sciences will use epithelial and smooth muscle cells, taken from the patient’s bladder, and layer them on to a synthetic tubular scaffold. The tube will then be surgically implanted inside the urethra.

The goal is for the progenitor cells to support self-renewal of the tissue and for the entire structure to become integrated into the surrounding tissue and become indistinguishable from it, restoring normal urinary function. Dr. Yoo and his team believe their approach has the potential to be effective for at least a decade.

“While not immediately life-threatening, urethral strictures lead to multiple health complications that impair quality of life and predispose to kidney dysfunction,” says Dr. Maria T. Millan, President and CEO of CIRM. “Developing an effective and durable treatment would significantly impact lives and has the potential to decrease the cumulative healthcare costs of treating recurrent kidney stones, infections and downstream kidney complications, especially of long-segment urethral strictures.”

First patient dosed in clinical trial for a drug-resistant form of epilepsy

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Tablet BM47753. Neo-Babylonian Period. Courtesy of the British Museum, London.

Epilepsy seems to have been a problem for people for as long as people have been around. The first recorded mention of it is on a 4000-year-old Akkadian tablet found in Mesopotamia (modern day Iraq). The tablet includes a description of a person with “his neck turning left, hands and feet are tense, and his eyes wide open, and from his mouth froth is flowing without him having any consciousness.”

Despite that long history, effective treatments for epilepsy were a long time coming. It wasn’t till the middle of the 19th century that physicians started using bromides to help people with the condition, but they also came with some nasty side effects, including depression, weakness, fatigue, lethargy, and coma.

Fast forward 150 years or so and we are now, hopefully, entering a new era. This week, Neurona Therapeutics announced they had dosed the first patient in their first-in-human clinical trial formesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy in adults. The trial specifically targets people who have a drug-resistant form of MTLE.

Neurona has developed a therapy called NRTX-1001, consisting of a specialized type of neuronal or brain cell derived from embryonic stem cells.  These cells are injected into the brain in the area affected by the seizures where they release a neurotransmitter or chemical messenger that will block the signals in the brain causing the epileptic seizures. Pre-clinical testing suggests a single dose of NRTX-1001 may have a long-lasting ability to suppress seizures.

A new approach is very much needed because current therapies for drug-resistant epilepsy are only partially effective and have serious drawbacks. One treatment that can significantly reduce seizure frequency is the removal of the affected part of the brain, however this can cause serious, irreversible damage, such as impacting memory, mood and vision.

CIRM has a vested interest in seeing this therapy succeed. We have invested more than $14 million over four different awards, in helping this research progress from a basic or Discovery level through to the current clinical trial.

In a news release, two key figures in administering the first dose to a patient said this was an important step forward. 

Harish Babu, M.D., Ph.D., assistant professor of neurosurgery at SUNY Upstate Medical University said: “Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy. Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue.”

Robert Beach, M.D., Ph.D. professor of neurology at SUNY Upstate Medical University added: “The objective of NRTX-1001 is to add cells that have the potential to repair the circuits that are damaged in epilepsy and thus reduce seizure activity.”

There is a huge unmet medical need for an effective, long-term therapy. Right now, it’s estimated that three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite dozens of approved drugs on the market.

If this therapy works it might mean that 4,000 year old tablet will become a medical footnote, rather than a reminder that we still have work to do.